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Sarepta Therapeutics Announces Positive Interim Results in Gene Therapy Trial for LGMD2E and Acquisition of Myonexus Therapeutics
On Feb. 27, Sarepta Therapeutics announced positive interim results of a phase 1/2a clinical trial for MYO-101, a gene therapy candidate developed by Myonexus Therapeutics for patients living with limb-girdle…
Buy a Shamrock, Grow Hope
It’s March, and at MDA that means only one thing: It’s Shamrocks season! Each St. Patrick’s Day season for the last 37 years, MDA has partnered with 20,000 retailers nationwide…
Tags: Fundraising, Shamrocks, Summer Camp
Wisconsin Sen. Tammy Baldwin Introduces Air Carrier Access Amendments Act
Last week, Sen. Tammy Baldwin of Wisconsin introduced the Air Carrier Access Amendments Act (S. 669). MDA commends Sen. Baldwin for championing this legislation that sets forth important protections for…
Clinical Trial Alert: ReveraGen BioPharma Seeks Participants for a Phase 2b DMD Study
Researchers at ReveraGen BioPharma are looking for ambulatory boys with Duchenne muscular dystrophy (DMD) to participate in a Phase 2b study. The goal of the study is to evaluate the…
FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease
On Feb. 25, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation (BTD) to AT-GAA, Amicus’ investigational combination drug therapy ATB200/AT2221 for treating late-onset Pompe disease. The…
Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study
Researchers at Amicus Therapeutics are looking for adults with late-onset Pompe disease (LOPD) to participate in a Phase 3 study. The goal of the study is to evaluate the safety…
In Their Shoes: 65 Years of IAFF’s Fight for MDA
In 1952 in South Boston, Charles Crowley was desperate for help. His two sons were living with Duchenne muscular dystrophy (DMD) and needed care. The family needed resources. Charles called…
Tags: Fill the Boot, Fundraising, Summer Camp
FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping
On Feb. 14, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) seeking approval under Priority Review for golodirsen (SRP-4053), its drug…
CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease
On Mar. 1, the Charcot-Marie-Tooth Association (CMTA) and the Muscular Dystrophy Association (MDA) announced that they have jointly awarded a research grant totaling $276,430 over three years to Kleopas Kleopa,…
Tags: Drug Development, Gene Therapy, Grants, Research
Strengthened by Spinraza, Marley Robinson is Tackling College — and Planning for More
Feb. 28 is Rare Disease Day, when the collective rare disease community raises awareness of the conditions with which we live and advocates for access to new novel treatments like…
Tags: Ambassadors, College, Education, Young Adults